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Stem Cell Transplant and Advanced Therapies

Unit 2 Lesson 2

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Student Learning Outcomes

At the end of the lesson, students will be able to:

Understand the principles and mechanisms
Explain potential impact on child growth and development
Identify the possible late effects of advanced therapies
Apply appropriate nursing management strategies

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Introduction

Stem cell transplants and T-cell therapies have revolutionized pediatric oncology treatment
They provide hope for children with relapsed or refractory malignancies

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Stem Cell Transplants

Principles and Mechanisms

Infuses healthy hematopoietic stem cells
Replaces damaged/diseased bone marrow
Goal

Restoration (after high-dose chemo/radiation)

Hematopoietic and immune system function

Stem cell sources

Autologous (client)
Allogeneic (matched donor)
Umbilical cord blood

(Cunha & Santos, 2021;

Diesch-Furlanetto et al., 2021)

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Stem Cell Transplants Process

High-dose chemotherapy and/or radiation

Eliminate diseased cells

Stem cell infusion

Regenerates healthy blood cells

(Dana-Farber Cancer Institute, n.d.)

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Types of Stem Cell Transplants

Autologous transplant

Client's own stem cells

Collected
Stored
Reinfused post high-dose therapy

Allogeneic transplant

Stem cells from matched donor

Examples

Sibling
Unrelated

(Diesch-Furlanetto et al., 2021)

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Types of Stem Cell Transplants continued…

Haploidentical transplant

Stem cells

Source

Partially matched donor

Usually a parent

Umbilical cord blood transplant

Stem cells

Source

After birth

Umbilical cord
Placenta

(Diesch-Furlanetto et al., 2021)

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Critical Thinking Question

The nurse is preparing to educate a family about their child's upcoming allogeneic stem cell transplant. What is the primary goal of this type of transplant?

a) To replace damaged or diseased bone marrow with the child's own stem cells

b) To restore normal hematopoietic and immune system function using donor stem cells

c) To collect and store the child's stem cells for future use

d) To regenerate healthy blood cells without the use of high-dose chemotherapy or radiation

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Impact on Growth and Development

Short Term Effects

Myelosuppression and associated complications

High-dose chemo/radiation

Causes

Neutropenia
Anemia
Thrombocytopenia

High risk for

Infections
Bleeding
Fatigue

(Diesch-Furlanetto et al., 2021)

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Impact on Growth and Development continued…

Acute graft-versus-host disease (aGVHD)

Allogeneic transplant recipients

Develop acute or chronic GVHD
Donor cells attack child's tissues

Can affect multiple organs

Skin
Liver
Gastrointestinal tract
Lungs

(Diesch-Furlanetto et al., 2021)

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Impact on Growth and Development continued…

Graft failure

Transplanted stem cells

Fail to engraft

Or are

Rejected

Lead to persistent pancytopenia
Require additional interventions

Symptoms: Mucositis and gastrointestinal symptoms

(Diesch-Furlanetto et al., 2021)

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Impact on Growth and Development continued…

Hepatic veno-occlusive disease (VOD)

Linked to several cancer therapies

Autologous
Allogeneic stem cell transplants

Occurrence

Regardless of underlying disease
Conditioning regimen type

(Diesch-Furlanetto et al., 2021; Kumar et al., 2003)

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Impact on Growth and Development continued…

Long Term Effects

Pulmonary complications

Obstructive
Restrictive lung diseases

Metabolic syndrome

(Diesch-Furlanetto et al., 2021; Kumar et al., 2003)

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Impact on Growth and Development continued…

Chronic graft-versus-host disease (cGVHD)

Skin

Rash
Scleroderma
Contractures

Oral cGVHD

Dry mouth
Taste changes
Dental problems

Pulmonary cGVHD

Bronchiolitis obliterans syndrome
Reduced lung function

(Diesch-Furlanetto et al., 2021)

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Impact on Growth and Development continued…

Growth and Development

Children

Achieve normal height
Low end of normal range

Total body irradiation (TBI)

Risk of slow or stunted growth

Growth hormone

Replacement therapy

Aid children aged ≤10 at transplant

Thyroid hormone deficiencies

Regularly monitor

(BMTinfonet.org, 2023)

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Impact on Growth and Development continued…

Puberty and Fertility

Boys

When transplanted before puberty

Often sterile
Especially with TBI or certain chemo drugs

Transplanted after puberty

Retain or recover fertility years later

Girls

Transplanted before puberty

Likely to retain fertility

Infants

Born to women who had childhood bone marrow transplant (BMT)

Healthy as those born to women without BMT

(BMTinfonet.org, 2023)

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Impact on Growth and Development continued…

Heart Health

Certain chemo drugs and TBI can

Damage heart muscle cells
Increases risk of later heart problems

Echocardiograms

Recommended every 2-5 years
With or without symptoms

(BMTinfonet.org, 2023)

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Impact on Growth and Development continued…

Dental Problems

High-dose chemo and/or TBI

Damages developing teeth
Delays eruption
Poor enamel/root formation
Tooth loss

Annual dental exams essential

(BMTinfonet.org, 2023)

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Impact on Growth and Development continued…

Learning and Cognitive Issues Post Transplant

Children

Learning difficulties

Especially with TBI or early transplant

Memory difficulty
Poor eye-hand coordination
Problem-solving challenges
ADHD

(BMTinfonet.org, 2023)

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Impact on Growth and Development continued…

Transitioning to Adult Care

Older children

Active participation in healthcare
Schedule appointments
Contact providers promptly
Manage healthcare needs
Access individualized survivorship care plan
Share with future providers

(BMTinfonet.org, 2023)

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Critical Thinking Question

The nurse is caring for a pediatric patient who received total body irradiation (TBI) before a stem cell transplant. What potential impact on growth and development should the nurse monitor for in this child?

a) Accelerated growth

b) Reduced risk of dental problems

c) Slow or stunted growth

d) Early onset of puberty

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Nursing Management

Infection prevention and control

Strict hand hygiene
Protective isolation
Environmental cleaning
Follow facility protocols
Monitor for signs of infection
Antimicrobial therapy

(Cunha & Santos, 2021)

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Nursing Management continued…

Symptom Management

Care for myelosuppression

Transfusions
Growth factors
Pain management

Manage GVHD symptoms

Immunosuppressants
Topical treatments
Supportive care

(Cunha & Santos, 2021)

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Nursing Management continued…

Client and Family

Educate

Transplant process
Post-transplant care

Medication adherence
Infection prevention
Lifestyle modifications

Potential complications
Long-term follow-up

(Cunha & Santos, 2021)

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T-Cell Therapies

Principles and Mechanisms

Target and destroy cancer cells

Process

T-cells collected through apheresis
Genetically engineered

Express chimeric antigen receptor (CAR)
Specific to tumor antigen

Expanded in laboratory
Reinfused into client

(Jones, 2024)

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T-Cell Therapies continued…

FDA-approved CAR T-cell Therapies

Tisagenlecleucel (Kymriah)

Children and young adults

Relapsed
Refractory B-cell acute lymphoblastic leukemia (ALL)

Recognized in May 2024
Only approved CAR-T therapy for pediatrics

Additional therapies

Approved for adults
In development

(Jones, 2024)

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Impact on Growth and Development

Short Term Effects

Cytokine release syndrome (CRS)

Symptoms

Fever
Hypotension
Hypoxia
Organ dysfunction

Causes

Rapid activation
Growth of T-cells

Effect

Massive release of inflammatory cytokines

(Jones, 2024)

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Impact on Growth and Development continued…

Short Term Effects continued…

Neurotoxicity

Manifestations

Encephalopathy
Seizures
Cerebral edema
Inflammation

Causes

Cytokines
Disruption of blood-brain barrier

(Jones, 2024)

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Impact on Growth and Development continued…

Short Term Effects continued…

B-cell aplasia

CAR T-cell therapy

Targets antigens on B cell surface
Destroys cancerous and normal B cells

Successful treatment

Results in low or absent B cells

Reduces ability to produce antibodies
Infection Prevention

Immunoglobulin replacement therapy

Intravenous
Subcutaneous

(BMTinfonet.org, 2023; Jones, 2024)

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Impact on Growth and Development continued…

Short Term Effects continued…

Tumor lysis syndrome

Discussed in Lesson 4 Unit 1

Risk of Infusion reactions

(Jones, 2024)

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Impact on Growth and Development continued…

Long Term Effects

Acute and Chronic Adverse Effects

Cytokine release syndrome (CRS)

Primary acute side effect

Symptoms

Range from mild to severe

Neurologic toxicities

Encephalopathy
Aphasia
Seizures

Reversible

(Callahan et al., 2019)

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Impact on Growth and Development continued…

Long Term Effects continued…

B-cell aplasia

Manageable with immunoglobulin infusions

Theoretical risks

Malignant transformation
T-cell lymphoproliferative disorders induction
Replication-competent retrovirus production

(Callahan et al., 2019)

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Impact on Growth and Development continued…

Long Term Effects continued…

Survivorship Considerations

Central and Peripheral Endocrine Dysfunction

High risk

Clients with TBI and allogeneic stem cell transplantation

Growth hormone and thyroid function

Monitor and intervene

(Callahan et al., 2019)

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Impact on Growth and Development continued…

Long Term Effects continued…

Gonadal Functioning and Fertility

Women and men

Infertile after TBI

Women

Hormone replacement for puberty

Men

Germinal cell dysfunction
Retain Leydig cell function

(Callahan et al., 2019)

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Impact on Growth and Development continued…

Long Term Effects continued…

Cardiac

TBI and anthracycline therapy have the highest risk of causing cardiac late effects

Ventricular dysfunction
Congestive heart failure
Arrhythmia
Dyslipidemia

(Callahan et al., 2019)

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Impact on Growth and Development continued…

Long Term effects continued…

Neurocognitive

Significant risk of learning disabilities

Causes

Intrathecal medication
High-dose antimetabolite therapy
TBI

Common problems

Decreased IQ
Slowed processing speed
Poor short-term memory
Concentration difficulties
Issues with executive function and organization

(Callahan et al., 2019)

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Impact on Growth and Development continued…

Long Term Effects continued…

Second Malignant Neoplasms

Risks before CAR T-cell therapy

Etoposide
TBI
High-dose cyclophosphamide
Cranial irradiation

Theoretical risk after CAR T-cell therapy

Malignant transformation of CAR T cells

(Callahan et al., 2019)

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Impact on Growth and Development continued…

Long Term Effects continued…

Psychosocial Implications

Families

PTSD
Financial distress
Psychosocial issues

Relocation
Separation from support networks

Siblings

Behavioral Issues
Sadness
Guilt
Anxiety
Isolation/abandonment
Jealousy

(Callahan et al., 2019)

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Impact on Growth and Development continued…

Long Term Effects continued…

Multidisciplinary approach essential

Physicians
Nurses
Social workers
Psychologists
Child life therapists
Creative arts therapists

(Callahan et al., 2019)

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Critical Thinking Question

The nurse is reviewing the long-term follow-up plan for a pediatric patient who underwent CAR T-cell therapy. What potential late effect should the nurse be aware of?

a) Increased risk of secondary malignancies

b) Improved neurocognitive function

c) Enhanced cardiac function

d) Spontaneous restoration of B-cell function and immunity

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Nursing Management

CRS and Neurotoxicity Management

Monitor closely
Administer biologic therapy ( Tocilizumab)
Corticosteroids for CRS management
Provide supportive care

Vasopressors
Oxygen therapy
Neurologic monitoring

(Jones, 2024)

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Nursing Management continued…

B-cell Aplasia Management

Monitor immunoglobulin levels
Administer IV immunoglobulin replacement
Educate children and families

Infection signs/symptoms
Prompt reporting

(Jones, 2024)

Contact info: info@nursesinternational.org

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Nursing Management continued…

Long Term Follow-up

Collaboration

Multidisciplinary team

Regular follow-up
Monitor potential late effects

Assess for development

Secondary malignancies
Endocrine disorders
Other long-term complications

Educate

T-cell therapy process
Potential complications

(Jones, 2024)

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General Nursing Management

Client and Family Education

Age-appropriate information

Treatment process
Side effects
Self-care measures
Medication adherence
Follow-up appointments
Preventive measures

Resources

Support groups
Counseling
Financial assistance

(Cunha & Santos, 2021)

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General Nursing Management continued…

Monitoring and Assessment

Vital signs
Pain
Other symptoms
Signs of infection

Fever
Chills
Redness at central line site

Signs of graft-versus-host disease

Skin rash
Diarrhea
Jaundice

Cytokine release syndrome symptoms

Fever
Hypotension
Respiratory distress

(Cunha & Santos, 2021;

Diesch-Furlanetto et al., 2021; Jones, 2024))

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General Nursing Management continued…

Medication Administration and Side Effect Management

Administer immunosuppressants

To prevent graft rejection or graft-versus-host disease

Provide supportive care medications

Antiemetics
Analgesics,
Growth factors

Monitor and manage medication side effects

(Cunha & Santos, 2021)

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General Nursing Management continued…

Emotional Support and Coping Strategies

Open communication
Expression of feelings
Age-appropriate activities

Interventions for normalization and distraction

Referrals to psychosocial services

Child life specialists
Social workers
Psychologists

(Cunha & Santos, 2021)

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General Nursing Management continued…

Multidisciplinary Collaboration

Physicians
Pharmacists
Dietitians
Other specialists

Multidisciplinary rounds
Care conferences

Facilitates communication and care coordination

Advocacy

Client's and family's needs
Preferences in the care plan

(Cunha & Santos, 2021)

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What Would the Nurse Do?

The nurse is caring for a child who recently underwent a stem cell transplant and is experiencing signs of acute graft-versus-host disease (aGVHD). What nursing management strategy should the nurse prioritize?

a) Encourage the child to engage in vigorous physical activity

b) Administer immunosuppressants as prescribed

c) Provide a high-fat, high-sugar diet

d) Limit visitation and social interaction

Contact info: info@nursesinternational.org

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References

BMTinfonet.org. (2023). Late Effects after a Bone Marrow Transplant

in Children.

https://bmtinfonet.org/transplant-article/late-effects-after-bone-marrow-transplant-children#

Callahan, C., Barry, A., Fooks-Parker, S., Smith, L., Baniewicz, D., &

Hobbie, W. (2019). Pediatric survivorship: Considerations following

CAR T-cell therapy. Clinical Journal of Oncology Nursing, 23(2), 35-41.

https://www.ons.org/cjon/23/2/supplement/pediatric-survivorship-considerations-following-car-t-cell-therapy

Contact info: info@nursesinternational.org

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References continued…

Cunha, I. C. K. O., & Santos, I. R. D. (2021). Nursing care for children

and adolescents submitted to hematopoietic stem cell

transplantation. Revista Gaúcha de Enfermagem, 42.

https://www.scielo.br/j/rgenf/a/pgw53C8FqXnTWcgG9rggSnn/

Dana-Farber Cancer Institute. (n.d.). Pediatric Stem Cell Transplant.

https://www.dana-farber.org/cancer-care/treatment/pediatric-stem-cell-transplant/about

Contact info: info@nursesinternational.org

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References continued…

Diesch-Furlanetto, T., Gabriel, M., Zajac-Spychala, O., Cattoni, A., Hoeben

, B.A.W. & Balduzzi, A. (2021). Late Effects After Haematopoietic

Stem Cell Transplantation in ALL, Long-Term Follow-Up and

Transition: A Step Into Adult Life. Frontiers in Pediatrics, 9:773895.

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8652149/

Jones, V. (2024). CAR T cell therapy for pediatric patients: What parents

should know. MD Anderson Cancer Center.

https://www.mdanderson.org/cancerwise/car-t-cell-therapy-for-pediatric-patients--what-parents-should-know.h00-159695967.html

Contact info: info@nursesinternational.org

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References continued…

Kumar, S., Deleve, L. D., Kamath, P. S., & Tefferi, A. (2003). Hepatic

Veno-occlusive Disease (sinusoidal obstruction syndrome) after

Hematopoietic Stem Cell Transplantation. Mayo Clinic Proceedings,

78(5), 589-598.

https://www.mayoclinicproceedings.org/article/S0025-6196(11)61929-0/fulltext

Leukemia & Lymphoma Society. (n.d.). Chimeric Antigen Receptor

(CAR) T-Cell Therapy.

https://www.lls.org/treatment/types-treatment/immunotherapy/chimeric-antigen-receptor-car-t-cell-therapy

Contact info: info@nursesinternational.org

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